RCPCH Royal College of Paediatrics and Child Health

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1 Turning the tide: five years on 5-11 Theobalds Road, London, WC1X 8SH RCPCH The Royal College of Paediatrics and Child Health (RCPCH) is a registered charity in England and Wales ( ) and in Scotland (SC038299). RCPCH Royal College of Paediatrics and Child Health

2 RCPCH Royal College of Paediatrics and Child Health Turning the tide: five years on Lindsey Hunter Anne Greenough Neena Modi

3 Acknowledgments We are grateful to the following colleagues who gave time and expertise to contribute to this report. Ms Emily Arkell Professor Helen Budge Dr Matthew Hyde Mr Martin McColgan Ms Angela Mensah Dr Andrew Prayle Dr Melanie Simpson We would also like to thank the research funders and organisations that provided us with data included in this report. Arthritis Research UK Biotechnology and Biological Sciences Research Council British Heart Foundation British Lung Foundation Cancer Research UK Diabetes UK Epilepsy Research Medical Research Council Medical Schools Council Meningitis Research Foundation National Institute of Health and Care Excellence National Institute of Health Research National Institute of Health Research Training Coordinating Centre Wellcome Trust

4 Foreword Paediatrics and child health can only advance by research. The stunning improvements in global child health over the last 15 years have all been based on research, some quite old. Child health has also improved in the UK over this period, and research leading to better practice has driven many of these changes. Several fields of paediatrics and child health have recently progressed very fast, others depressingly slowly. In general these mirror the rate of scientific advance. In all fields patients who are enrolled in clinical research tend to do better than those who are not. It is therefore a moral as well as a scientific responsibility to maximise the strength of research for children in the NHS, and to ensure children have the opportunity to be involved in research if they have health problems for which optimal treatment has not yet been determined. The initial RCPCH report in 2012, Turning the Tide, was extremely influential in highlighting the opportunities but also the limitations of neonatal, paediatric and child health in the UK, and galvanised funders to look again at their models. This report follows up on that analysis 5 years later, and again examines the state of UK research and the academic base to see what has changed. Some things have improved, including several new funding streams for research in and with children. Several objective metrics, such as publications by paediatric specialists have increased. There are however some worrying trends, and in particular the decrease in academic paediatricians at senior lecturer grade, and the limited amount of time (often zero) that paediatricians of all grades have for research in their job plans. Arguably too little research is taking place relative to other areas of medicine. A discipline that chooses not to prioritise research is choosing not to advance, and it is therefore strongly in the interests of children in the UK that the RCPCH, and its outgoing President Professor Modi, have highlighted this issue so clearly. There are clear data-driven messages for many groups in this report: for funders of research; for scientists and clinical researchers; for practicing paediatricians and their employers. We all need to take the messages from the data presented here seriously, and act on it. Professor Chris Whitty Chief Scientific Adviser Department of Health and Social Care

5 Executive summary In December 2012, the Royal College of Paediatrics and Child Health (RCPCH) launched Turning the Tide, a report that shone a spotlight on the importance and relevance of biomedical and life sciences research focussed on the needs of infants, children and young people. Over the ensuing five years there have been many developments that highlight a national commitment to sustaining and strengthening UK basic science and clinical research, and the life sciences industries. These include the 100,000 Genomes Project, and the establishment of UK Research and Innovation, Innovate UK, and Health Data Research UK. Paediatricians continue to advance the evidence-base for practice, the National Institute for Health Research provides a globally unparalleled framework for the delivery of clinical trials involving children, and the UK Research Councils and charities have provided sustained funding opportunities. We welcome these developments and these commitments. There are however, areas that must not be forgotten. A poor evidence-base for practice is a patient safety issue. Strong basic science and clinical research are essential to driving improvement, testing treatments, reducing uncertainties in patient care and evaluating the effectiveness of innovations. The uncertainties of the planned departure of the UK from the European Union and the severe pressures currently facing UK health services may limit opportunities for trainees and the involvement of clinicians in research. The paediatric academic workforce remains small. There is inadequate representation of children s interests in the UK life sciences strategy and few paediatricians on national research boards and committees. Reliable paediatric and related data sources are growing in number and improving in quality, as is child centred research infrastructure, but more could be done to integrate development and advance the pace of change. Research in crucial areas such as public health continues to be limited. The RCPCH is committed to strengthening basic science and clinical research, and the development of devices, medicines and technologies that address the needs of children. We wish to see paediatric practice driven by the excitement of discovery science, and the curiosity and questioning that are at the heart of good medicine. We will sustain and improve education and training in research skills for all clinicians caring for children, and support paediatricians who wish to pursue an academic career. We will continue to advocate for child health expertise and the perspectives of the paediatric population to be represented on national research boards and committees. We aim to grow our engagement with industry and promote opportunity for the involvement of paediatricians to contribute their expertise to the development and evaluation of products. Good science does not stand still, so we will track progress over time to ensure

6 that infants, children and young people, and so too, UK population wellbeing and prosperity, benefit from a sustained focus on strengthening child health research.

7 Abbreviations ACF BRC BRU CCT CL COiN CRF CRN EU HDR-UK HRA IPF MCRN MHRA NIHR OOPR PA PPI RCPCH RCT SASS StaR TITCH UKCHRC UKRI Academic Clinical Fellowship Biomedical Research Centre Biomedical Research Unit Certificate of Completion of Training Clinical Lecturer Core Outcomes in Neonatology Clinical Research Facility Clinical Research Network European Union Health Data Research UK Health Research Authority In-Practice Fellowship Medicines for Children Research Network Medicines and Healthcare products Regulatory Agency National Institute for Health Research Out of Programme (Research) Programmed Activity Public Patient Involvement Royal College of Paediatrics and Child Health Randomised Controlled Trial Staff, Associate Specialist and Specialty Standards for Research Technology Innovation Transforming Child Health UK Child Health Research Collaboration UK Research and Innovation

8 Contents 1 Introduction Changes in the UK biomedical research landscape Child health research activity, outputs and funding The paediatric clinical academic workforce Paediatric representation on research boards and committees Involvement of children, young people and families in research RCPCH actions Conclusions References... 57

9 1 Introduction In 2012, the Royal College of Paediatrics and Child Health (RCPCH) published Turning the Tide [1, 2], a report on children s clinical and biomedical research. This covered training, capacity, infrastructure and outputs, and brought a spotlight to bear on the importance of clinical and biomedical research in improving the health and wellbeing of children. In Turning the Tide the RCPCH made several recommendations aimed at improving earlyyears research, and raised the need for strengthened: Education, training and guidance, to assist the child health workforce in supporting, delivering and leading clinical research Engagement with children, young people and families, to hear their voices and how they want to be involved in and benefit from research Collaboration, between organisations to coordinate efforts to fund high cost projects, programmes, infrastructure and training, and raise the profile of child health research Capacity, by increasing the number of clinical and non-clinical child health researchers and establishing child-appropriate infrastructure Integration, of children s research with preclinical science and clinical practice Five years on from Turning the Tide, we evaluate progress, reflect on our own actions, and identify next steps. 1

10 2 Changes in the UK biomedical research landscape Since the publication of Turning the Tide [1, 2], several changes have taken place within UK healthcare research systems, and the country is currently planning for a future outside the European Union (EU). The full implications of leaving the EU are unknown and may have a challenging impact on child health research. In this changeable environment, it is a priority of the RCPCH to continue to promote the rights of the child to high quality, evidence-based healthcare and to strengthen the underpinning scientific basis of paediatric practice. 2.1 NIHR Clinical Research Networks The Medicines for Children Research Network (MCRN) was established in 2005, funded by the Department of Health as part of the National Institute of Health Research (NIHR) Clinical Research Network (CRN). The Paediatrics (non-medicines) Specialty Group was subsequently formed in 2007 and together with the MCRN helped drive improvements in paediatric clinical research in the UK. In 2014, changes to the structure of the CRN led to the amalgamation of these two elements to form the National Children s Speciality [3]. This is one of 30 CRN groupings bringing together communities of clinical practice in national networks of research expertise. The Children s Specialty leads, manages and supports a portfolio of research studies related to children. These initiatives have been very successful, resulting for example in a large number of children recruited to clinical trials and the UK participating in the highest number of pan- European clinical trials for childhood diseases, facilitated by harmonised frameworks for conducting studies [4]. In the future, developments in precision medicine and more targeted treatments will mean that many trials will increasingly be applicable to smaller national patient cohorts and hence rely even further on coordinated networks [5]. 2.2 The UK Life Sciences Industrial Strategy The UK continues to prioritise the development of the life sciences sector as a major component of the national economic base. The life sciences industries involve the application of biology and technology to health improvement, and include biopharmaceuticals, medical technologies, genomics, diagnostics and digital health. The life sciences industrial strategy published in August 2017 [6] set out a vision of how the UK might exploit existing strengths to increase the pace of economic growth in this sector, emphasising the necessity for sustained effort over a long period if the UK is to maintain a competitive edge. The strategy is based around nine core recommendations i) creating a Health Advanced Research Programme to undertake large research projects 2

11 and create new high-risk healthcare industries; ii) increasing funding for basic science; iii) continuing to improve UK clinical trial capabilities; iv) a tax environment that supports growth; v) creating Life Sciences clusters, a single front door to the UK for research collaboration, partnership and investment; vi) attracting investment; vii) adopting and advancing the Accelerated Access Review to speed the delivery of new products to patients; viii) establishing Digital Innovation Hubs; ix) developing and delivering an action plan based on a gap analysis of key science skills. The UK Life Sciences Industrial Strategy provides no specific reference to infants, children and young people. However, research targeting these groups is essential to improve understanding of childhood disease as well as the developmental origins of adult health and disease. The growing burden of long-term conditions such as obesity, cardiovascular disease, vascular dementia and diabetes have a substantial component of their origins in early life and are adversely affecting the health and economic wellbeing of the nation [7]. A focus by the life sciences sector upon life-course research has substantial potential to benefit patient and population health, and hence reduce NHS costs and improve adult productivity and the UK economy. The UK has undoubted strengths to support life-course research, including a unified health system, ability to link whole population datasets, and mature clinical networks. The RCPCH considers it important that the UK recognise the benefits of the involvement of child perspectives in strategic plans for the life sciences. The development by the RCPCH of a framework for interaction with industry will facilitate the development of stronger links and promote transparent collaborations with the aim of benefiting child and population health [8]. The RCPCH are also pleased to note the launch in 2017 of the UK Prevention Research Partnership, an alliance of UK charities, Research Councils and the Health Departments to supports research that targets population-level health, especially the physical and social antecedents of non-communicable diseases. 2.3 A UK-wide framework for Health and Social Care Research In October 2017, the Health Research Authority (HRA) published the UK policy framework for Health and Social Care Research [9], replacing the separate Research Governance Frameworks previously in place for each nation. The framework sets out principles of good practice for the management and conduct of health and social care research in the UK, bringing together the Health Research Authority and the health departments in Northern Ireland, Scotland and Wales, and facilitating the conduct of research across all UK nations. The new framework outlines the responsibilities of organisations undertaking health and social care research, aims to streamline management and conduct across the UK, and 3

12 promotes the interests of patients. Recent changes include the assessment of Walesonly non-commercial studies through the NIHR Clinical Research Network noncommercial adoption process, emulating existing arrangements in place for Scotlandonly studies. The principal justification for adoption is that a study is of clear value in meeting the priorities, needs and realities of the NHS. The RCPCH welcomes this framework, as alignment of operational processes across the UK nations is an important facilitator of research. The RCPCH would also welcome the inclusion of infants, children and young people as the default in research studies unless this can clearly be shown to be unnecessary or inappropriate, and has made representations to this effect to the HRA though the National Research Advisory Panel. 2.4 UK Research and Innovation There is high level recognition of the need for the UK to keep research and innovation at the forefront of national endeavour. A component of the Higher Education and Research Bill (part 3) proposed the establishment of UK Research and Innovation (UKRI) [10]. UKRI will launch in April 2018 and operate across the UK with a budget of 6 Billion. UKRI will bring together the seven research councils, Innovate UK and a new organisation, Research England, in partnership with equivalent organisations in the devolved nations, to provide a strong voice for research and innovation to government and internationally, to ensure the UK maintains its position as a world leader. UKRI will be measured by the impact delivered in relation to three elements, i) extending the frontiers of human knowledge and understanding; ii) delivering economic impact and social prosperity; and iii) supporting society to become enriched, healthier, more resilient and sustainable. UKRI will address science and technology as well as the social sciences, arts and humanities and will be responsible for allocating 4.7 Billion from the 2016 Autumn Statement. The RCPCH will continue to reiterate the importance to population health and the national economy of a strong focus on research to improve child health and wellbeing. 2.5 Innovate UK Innovate UK is the operating name of the Technology Strategy Board, an arm s length body reporting to the Department for Business, Energy and Industrial Strategy. The name Innovate UK was adopted in August Innovate UK [11] drives productivity and growth by supporting businesses to realise the potential of new technologies, develop ideas and make them a commercial success. Innovate UK promotes growth by funding science and technology development, linking innovators to partners and helping them launch and grow successful businesses. In the coming years, as Innovate UK becomes the business- 4

13 facing part of UKRI, the aim is to lead on delivering economic impact and creating jobs. Innovate UK will invest around 500 Million in the financial year 2017/18 in competitions supporting business-led innovation and in innovation infrastructure that allows businesses to access leading-edge expertise, equipment and facilities. The RCPCH is involved in discussions with Innovate UK around mechanisms to connect child health expertise and knowledge with industry partners. 2.6 Health Data Research UK Informatics provides a powerful approach to improving the monitoring of population health and prevention, providing novel insights into disease pathways and patient care, and increasing the efficiency and effectiveness of health and biomedical research. Health Data Research UK (HDR-UK) is a multi-funder UK institute for health and biomedical informatics research [12]. This national, interdisciplinary research institute will capitalise on the renowned data resources and research strengths of the UK, developing the capacity and methods to accelerate the pace and scale of health and biomedical data science to deliver a step change in UK capabilities. The institute will be delivered in partnership with the health research departments of England, Scotland and Wales, the Economic and Social Research Council and the Engineering and Physical Sciences Research Council, Wellcome Trust, and British Heart Foundation. A number of other charity, government and industry partners have also pledged their support and it is anticipated that additional organisations will participate in this ambitious endeavour. The RCPCH would like to see the development and capture of child health datasets and inclusion of these in HDR-UK. 2.7 Improvement Research Institute The University of Cambridge is developing a new Improvement Research Institute, supported by 40 Million funding from the Health Foundation over a period of 10 years. Health improvement research builds and utilises knowledge to improve the quality of health services for the benefit of patients and populations. The new Institute aims to strengthen the evidence base for how to improve patient care, produce practical highquality learning, and build research skills in the NHS and academia, as well as working with partners within the wider research and improvement communities, NHS staff, patients and carers to identify, design and test improvements. The Institute will also fund fellowship programmes to build skills in improvement research in the UK. The RCPCH welcomes the development of the Institute, and hopes to see representation of the needs of infants, children and young people in their work. 5

14 2.8 Twelve actions to support research in NHS England In April 2017, NHS England set out initial goals to support research. Following this NHS England, NIHR, the Office for Life Sciences, the HRA and other partners outlined 12 actions to support and apply research. These centre on simplifying NHS research processes, articulating NHS research priorities, enhancing data infrastructure, supporting advanced research in cutting-edge technologies, and improving and simplifying adoption ecosystems. NHS England, with NIHR, Office for Life Sciences, HRA and other partners, will implement these actions [13]. Clinicians are central to the successful delivery of patient-centred research, particularly studies involving infants, children and young people, where expert knowledge and skills are required. The RCPCH also considers it essential to incorporate the views and priorities of patients throughout the research pipeline. The RCPCH therefore wishes to draw attention to the necessity for close collaboration between NHS England, NHS Employers, Health Education England, NIHR and patient groups, to ensure consultant job-plans reflect their contribution to the delivery of research studies, capture of high quality clinical data, and the acquisition of generic research skills during the training of doctors, nurses and allied health professionals. 2.9 Improving the quality of paediatric research A number of initiatives are underway to improve the quality of paediatric research. Standards for Research (StaR) in Child Health [14] was founded in 2009 and involves methodologists, clinicians, patient advocacy groups and policy makers in developing practical, evidence-based standards for enhancing the reliability and relevance of paediatric clinical research. Core Outcomes in Neonatology (COiN) is a project initiated in 2015 to develop a neonatal Core Outcomes Set based upon data routinely recorded during clinical care and held in the National Neonatal Research Database. This will ensure that outcomes of importance to all stakeholders, including patients and parents, are reported in a standard way that is relevant to everyday neonatal care. Embedding a core outcomes set into clinical datasets will enhance the usefulness of routine information in informing practice, enhancing patient care and improving outcomes The EU (Withdrawal) Bill The departure of the UK from the EU has potential to have a profound influence on many aspects of biomedical research, including the pharmaceutical and medical device industries, regulation of medical products, and the underpinning UK preclinical and clinical research base. The RCPCH has been observing developments closely and has 6

15 been pleased that at the end of 2017, confirmation was issued that UK institutions can continue to bid for EU Horizon 2020 funding post-brexit, in 2019 and 2020 [15]. However, clearly many uncertainties remain. A new Clinical Trial Regulation, which will streamline approval processes for international trials and create a new registration portal for all studies conducted in the EU was passed in 2014 but has not yet been implemented (Regulation (EU) No 536/2014) [16]. The European Medicines Agency Management Board has confirmed the intention that the Clinical Trials Regulation will be outside the scope of the EU (withdrawal) Bill (Letter from the Department for Exiting European Union, 12 October 2017) [17] but it is unclear how this will be interpreted by the UK, particularly in a potential implementation period Children s medicines development Historically, variation in legislation and standards presented a significant obstacle to developing medicines for children. Additionally, infants, children and young people have long been recognised to be served poorly by market forces in relation to the development and supply of medicines. Paediatric considerations have been an integral part of pharmaceutical research and medicines development across the EU in recent years [18]. The specific needs of infants, children and young people were recognised in the European Regulation No 1901/2006 on medical products for paediatric use. The Paediatric Regulation, implemented in 2007, received extensive UK input. It provided pan-european incentives to the pharmaceutical industry to develop paediatric products. This had a positive impact, as discussed in the 2016 European Medicines Agency 10-year Report to the European Commission [18], resulting in more than 10,000 paediatric investigation plans, and a decrease in off-label use of adult medicines in infants, children and young people. However, the report identified little progress in treatments for diseases that only affect infants, children and young people, or where conditions, particularly rare diseases, show biological differences between adults and children. Overall, however, the benefits of the Paediatric Regulation to-date, in respect of children s medicines development, have outweighed any related financial or administrative burdens. With the UK exiting the EU there is growing concern that this framework and the standing of the UK in relation to children s medicines development will be lost, and that the pace of reduction in the continuing high usage of off-label and unlicensed medicines in children will be halted. Further, if research in the UK is not completely aligned with the European Paediatric Regulations, the volume of commercial research in the UK will be reduced with strong negative impacts on child health, the NIHR portfolio, and the UK life sciences industries. 7

16 European regulatory frameworks for licencing medicines have been instrumental in speeding access to products by creating a larger market in which to launch new drugs, and facilitating post-licensing surveillance using evidence from across the EU. Harmonised regulation facilitates international collaboration including clinical trials involving children, and rare diseases, where eligible patient numbers can be small. There have been considerable benefits to the UK from being a member of the European Medicines Agency. The costs and burdens of developing and supplying paediatric medicines have been minimised as the UK has been embedded in a strong international collaboration that includes the free-flow of medicines and shared international standards. The UK does not have the capability to work as a standalone regulatory jurisdiction in paediatrics and does not have sufficient market size to justify commercial investment in UK-only development programmes for infants, children and young people. If the UK Medicines and Healthcare products Regulation Agency (MHRA) is to become a sovereign regulator, operating outside of the European framework, it will need to increase capability and capacity rapidly to manage the increased workload and develop strategies to overcome the diseconomies of scale arising from the relatively small UK population of children, especially those with rare conditions. The RCPCH has strongly advocated that any post-brexit arrangements with respect to the development of medicines take into account the specific needs of children. Whatever the future arrangements, a priority must be for UK infants, children and young people to have timely access to medicines that have been evaluated rigorously and in a standard way. They will best be served by the strongest possible involvement of the UK with the European medicines regulatory network, the European Medicines Agency and opportunity for involvement in pan-european studies. For these reasons, the RCPCH has called for the UK to maintain a close working relationship with the European Medicines Agency and that paediatric capability at the MHRA is expanded as necessary to meet the challenges of a post-brexit era ,000 Genomes Project The 100,000 Genomes Project aims to bring the benefits of personalised medicine to the NHS. It involves a UK Government commitment to sequence 100,000 human genomes by the end of NHS England has established 13 NHS Genomics Medicine Centres to deliver an end-to-end genomic medicine pathway across the country. They obtain informed consent from patients and family members, collect samples for DNA extraction and sequencing, capture clinical information and establish infrastructure to make genomic medicine a routine part of NHS care. Training is an integral part of the long-term plan to incorporate genomic information into mainstream clinical medicine. Health Education England runs a Genomics Education Programme comprising short and 8

17 postgraduate courses, and other resources for doctors and healthcare workers. There is also a programme to educate the public about genomics and its implications. The RCPCH is pleased that neonates will be among the first wave of patients to benefit from the commissioning of genomic sequencing Medical technology initiatives There is increasing opportunity to improve patient care through the use of technology. A number of medical-technological initiatives have been introduced to identify and tackle unmet needs and improve clinical practice. Technology Innovation Transforming Child Health (TITCH) is national network dedicated to the development of technology solutions to improve children s healthcare. TITCH brings together a number of children s hospitals and specialist centres, medical technology co-operatives (centres of expertise that work with industry to develop new medical device concepts to improve treatment and quality of life for patients), smallmedium enterprises, academic partners and charities. TITCH provides a platform to facilitate the development and commercialization of innovations for the benefit of children and families. To date TITCH has worked with the NIHR Med-Tech Co-operative Devices for Dignity and leveraged approximately 5.5 Million to support the development of health technologies for children and young people. This included a funding call supported by TITCH as part of the NHS England Small Business Research Initiative in focusing on self-care and independence in children with long-term conditions. In April 2017, TITCH and the Northern Health Science Alliance hosted a workshop Bridging the Gap, bringing together clinical experts, industry experts, designers, engineers and academics to discuss barriers to the adoption and implementation of technology for child health. Following on from this the TITCH Network and the Children s Clinical Research Network brought together multiple high-level stakeholder representatives to determine approaches to accelerating child health technology development and adoption in the NHS. In early 2018, TITCH will support the Small Business Research Initiative in the funding call for Child and Adolescent Mental Health. Sheffield Children s Hospital hosts the NIHR Children and Young People MedTech Cooperative established to support the development of technology specifically for child health, supported by a 1.1 Million grant from the NIHR in Sheffield Children s Hospital will work as a centre of expertise in collaboration with other children s healthcare Trusts across the country bringing together patients, clinicians, researchers, commissioners and industry to focus on themes that include epilepsy, muscle disorders, respiratory conditions, ventilation in children, sleep disorders, childhood cancer, rare diseases, transition and paediatric surgery. 9

18 The Children s Research Industry Group facilitates collaboration with industry, linking the NIHR children s research network and other organisations with representatives in industry working on children s medicines, devices and other areas. The aim is to improve collaboration and the support offered to industry particularly during early programme development, facilitate the NIHR children s research network and other affiliated organisations to obtain advice on industry matters, and attract additional studies to the UK. GOSgene was established in 2010 funded by the NIHR Biomedical Research Centre at Great Ormond Street Hospital and University College London to improve gene identification in uncharacterised diseases, diagnostic testing, genetic counselling, and functional analyses aimed at understanding pathogenesis, improving patient management and developing new therapies. Collaborations have been built with industry through links with the North-East Thames Regional Genetics Service. The Leeds in vitro diagnostics co-operative is a partnership between Leeds Teaching Hospitals NHS Trust and the University of Leeds. The co-operative acts as a catalyst for the generation of high quality evidence on clinical validity, utility, cost effectiveness and benefits of commercially-supplied tests, fosters collaboration between industry, healthcare professionals, patients, commissioners and researchers, creates new, world class methodologies for the assessment of in-vitro diagnostics and engages with patients to shape strategies and identify new opportunities Pregnancy Research Review Group This specialist group was established following the annual report The Health of the 51%: Women from the UK Chief Medical Officer in 2014 [19]. The Group has multiorganisational representation that includes the RCPCH. It was established through the UK Clinical Research Collaboration to develop strategic approaches in priority areas of pregnancy research and work with research funders to review needs and spend. Particular research areas highlighted include pre-conception interventions to improve maternal and child mental and physical health, screening tests, prevention and treatment of pre-eclampsia, fetal growth restriction and preterm birth, and optimum models for antenatal and postnatal care. The remit of the group also includes the development of strategies to promote the UK as an excellent environment for pregnancy research given the unique ability of the NHS to undertake long-term follow-up at scale, and develop and foster academic-nhs-industry links. 10

19 2.15 Conclusions There have been many developments over the last five years in the UK life sciences, and in clinical research organisation and regulation. These highlight the drive and commitment to develop and strengthen biomedical research in the UK. The inclusion of a focus on infants, children and young people offers opportunity for wide-ranging, longterm benefit, particularly in areas such as life-course research which requires multidisciplinary collaboration and is facilitated by integrated health systems such as exist in the UK. This, together with the uncertainties posed by Brexit, provides strong justification for continued drive to strengthen UK infrastructure for children s research, increase the involvement of paediatric expertise in discovery science, and advocate for regulatory frameworks harmonised with international partners and appropriate for child populations. 11

20 3 Child health research activity, outputs and funding 3.1 Children s medical research facilities in the UK Biomedical Research Centres (BRC) and Biomedical Research Units (BRU) were established in 2008 to carry out research for the benefit of patients. They are funded by the NIHR and based within the NHS and universities. In 2012, 11 BRC and 20 BRU were funded to lead the translation of biomedical research into clinical research. Only one BRC was child focused, and no BRU. The NIHR 2017 funding round combined funding for centres and units under the title of BRC (Table 1). Twenty BRC were funded in England, only one of which is exclusively focused upon children (Great Ormond Street Hospital for Children and University College, London), although others have a child focus within adult-centred core themes. Clinical Research Facilities (CRF) are dedicated facilities, where specialist clinical research and support staff from universities and NHS Trusts work together on patientorientated commercial and non-commercial experimental medicine studies. There are 19 NIHR funded CRF, eight of which receive additional funding from other sources or with NIHR funding routed through Trusts (e.g. Great Ormond Street Hospital for Children and Sheffield Children s NHS Foundation Trust). 12

21 Table 1 NIHR Biomedical Research Centres Organisation Academic partner Research themes 1 Barts Health NHS Trust Queen Mary University of London Cardiovascular devices and innovative trials, inherited cardiovascular disorders 2 Cambridge University Hospitals NHS Foundation Trust University of Cambridge Antimicrobial resistance, cancer, cardiovascular and respiratory disease, dementia and neurodegenerative disease, gastrointestinal disease, integrative genomics, mental health, metabolism, endocrinology and bone, neuroscience, nutrition, diet and lifestyle, population and quantitative science, transplantation and regenerative science, women s health and paediatrics 3 Central Manchester University Hospitals NHS Foundation Trust University of Manchester Advanced radiotherapy, cancer prevention and early detection, cancer precision medicine, dermatology, hearing health, respiratory medicine, targeted therapy in musculoskeletal diseases 4 Great Ormond Street Hospital for Children NHS Foundation Trust University College London Advanced treatments for structural malformation and tissue damage, gene, stem and cellular therapies, genomics and systems medicine, novel therapeutics and their translation into childhood disease 5 Guy s and St Thomas NHS Foundation Trust King s College London Cardiovascular disease, cutaneous medicine, genomic medicine, imaging sciences, infection and immunity, oral health, regenerative medicine and cellular therapy, transplantation, women and children s health 6 Imperial College Healthcare Trust Imperial College London Brain sciences, cancer, cardiovascular, gut health, immunology, infection and AMR, metabolic medicine and endocrine, surgery and technology 7 Leeds Teaching Hospitals NHS Trust University of Leeds Preventing disease and disability in immune mediated inflammatory disease, improving treatment of osteoarthritis 8 Moorfields Eye Hospital NHS Foundation Trust University College London Gene therapy, genomic medicine and informatics, inflammation and immunotherapy, regenerative medicine and pharmaceutics, visual assessment and imaging 13

22 9 Newcastle upon Tyne Hospitals NHS Foundation Trust 10 Nottingham University Hospitals NHS Trust 11 Oxford Health NHS Foundation Trust 12 Oxford University Hospitals NHS Foundation Trust 13 Royal Marsden NHS Foundation Trust 14 Sheffield Teaching Hospitals NHS Foundation Trust 15 South London and Maudsley NHS Foundation Trust 16 University College London Hospitals NHS Foundation Trust 17 University Hospitals Birmingham NHS Newcastle University Dementia, liver disease, musculoskeletal disease, neuromuscular disease, skin and oral disease University of Nottingham Deafness and hearing problems, gastrointestinal and liver disorders, mental health and technology, musculoskeletal disease, respiratory disease University of Oxford Adult mental health, older adults and dementia, precision psychological treatments University of Oxford Antimicrobial resistance and microbiology, cardiovascular, diabetes and metabolism, gastroenterology and mucosal immunity, genomic medicine, haematology and stem cells, multi-modal cancer therapies, multi-morbidity and long-term conditions, musculoskeletal, neurological conditions, obesity, diet and lifestyle, respiratory, stroke and vascular dementia, surgical innovation and evaluation, technology and digital health, vaccines for emerging and endemic diseases Institute of Cancer Research Breast cancer, gastrointestinal cancers, novel cancer therapeutics, prostate cancer, targeted physical therapies, uncommon cancers University of Sheffield Translational neuroscience for chronic neurological disorders King s College London Affective disorders and interface with medicine, bioinformatics and statistics, biomarkers and genomics, child and neurodevelopmental disorders, clinical and population informatics, dementia and related disorders, mobile health, neuroimaging, obesity, pain, patient and carer involvement and engagement, psychosis and neuropsychiatry, substance use, translational therapeutics University College London Cancer, cardiovascular disease, deafness and hearing, dementia and mental health, immunity, inflammation and immunotherapeutics, neurological diseases, obesity, oral health and disease University of Inflammatory arthritis, inflammatory bowel disease, inflammatory sarcopenia 14

23 Foundation Trust Birmingham 18 University Hospitals Bristol NHS Foundation Trust University of Bristol 19 University Hospitals of Leicester NHS Trust University of Leicester 20 University Hospital Southampton NHS Foundation Trust University of Southampton Cardiovascular disease, mental health, nutrition, diet and lifestyle (including obesity), reproductive and perinatal health, surgical innovation Cardiovascular, lifestyle, respiratory Life-course nutrition, lifestyle and health, respiratory and critical care 15

24 3.2 Child health research activity Review of the NIHR Portfolio shows how activity has changed over time. Over the period 2010/11 to 2016/17 there has been a mean annual increase of 6.2% (202) in studies adopted onto the Children s Speciality Portfolio, including an increase in commercial studies (Figure 1) 1. In 2012, the Children s Speciality Portfolio represented 2.9% of the total number of open studies across the NIHR CRN portfolio, but by 2015 this had risen to 7.6%. The NIHR open data platform shows that approximately 460,000 participants have been recruited to studies in the Children s Specialty Portfolio since 2010/2011 (Table 2). There are many other examples of strong involvement of children in clinical trials. For example, the Oxford Vaccine Group has enrolled over 10,000 children and young people into phase II, III and IV clinical trials since 2001 [20]. Some of the participants recruited to studies in the Children s Specialty Portfolio may be parents or carers and some studies that involve children, for example in cancer and child mental health, are not led by the Children s Specialty, so that the exact number of children recruited and their age distribution are unknown. Going forward, the RCPCH suggests that recording age or year of birth of all study participants would address this information gap. Table 2 Annual UK recruitment to studies on the NIHR CRN Children's portfolio* Year 2010/ / / / / / /17 Total Total recruitment 39,643 57,266 71,296 80,043 83,727 62,571 65, ,587 *Studies led and supported by the Children's Portfolio 1 The NIHR financial year runs from April to March. 16

25 Figure 1 Number of commercial and non-commercial studies adopted onto the NIHR Children s Speciality Portfolio by year [Data provided by the NIHR Children s Speciality Portfolio, obtained from NIHR Open Data Platform] 3.3 Registered children s clinical trials A systematic search carried out by Pandolfini and Bonati [21] in 2009 identified clinical trials involving children across nine registries. A similar search was undertaken for the 2012 Turning the Tide report [1] and repeated in The 2012 and 2017 searches found an additional 11 registries that were not included in the 2009 Pandolfini and Bonati publication. A total of 14.0% of registered trials were identified as being conducted in the paediatric population in 2017, compared to 14.6% in (data from the ReBec registry was not included as it does not provide an accurate figure for the number of paediatric trials). Although this percentage has remained essentially unchanged, there has been an increase in the absolute number over the last five years, from 75,854 paediatric clinical trials registered in 2012 to 116,363 in This equates to a 61% increase in paediatric clinical trials undertaken worldwide (Table 3). Trends in the percentage of trials that are 2 Some caution is required in interpreting the raw data presented in table 3 for individual registries, as many studies are entered into multiple databases. 17

26 paediatric are however inconsistent across registries. In some regional registries, such as the Australian and New Zealand Clinical Trials Registry, the percentage has increased, but in the largest trial database, Clinicaltrials.gov, the percentage has consistently decreased since The extent to which the increase in the absolute number of registered paediatric clinical trials represents a genuine increase as opposed to improved registration or variability in categorisation is also unclear. 18

27 Table 3 Clinical trials registered in trial databases across the world, and the percentage recruiting participants aged <18 years (searches conducted August 2012 and August 2017) Register Remit of Register Number of paediatric trials (2017) Number of trials in register (2017) % of paediatric trials (2017) Number of paediatric trials (2012) Number of trials in register (2012) % of paediatric trials (2012) % of paediatric trials (reported in 2009) Search strategy AMGEN ( en.com) Separate register now subsumed by clinicaltrials.gov. Link on the AMGEN website allows search of clinicaltrials.gov for registered pharmaceutical trials being conducted by AMGEN Clinicaltrials.gov searched using Amgen as sponsor and age limit <18 years ANZCTR ( tr.org.au/) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in the Australasia Age limit <18 years Chinese Clinical trial Registry ( r.org/en/) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in the China Searched the following terms individually, then excluded duplicates: child, infant, paediatric, pediatric, adolescent, toddler, baby, babies, 19

28 Clinicaltrial s.gov India- Clinical Trials Registry (ctri.nic.in) ISRCTN ( rolledtrials.com/ ) MRC CTU ( mrc.ac.uk/ study_sear ch.aspx) Hospital, university and pharmaceutical research. Observational and interventional studies. Open to all research globally. Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in the India. NHS, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in the UK. Mainly RCT currently being undertaken by the MRC clinical trials unit. neonate, newborn Age limit <18 years Searched the following terms individually, then excluded duplicates: paediatric, pediatric, child, children, kids, adolescent, infant, newborn, toddler, baby, babies, neonate Searched using the following strategy: pediatr% OR paediatr% OR adolesc% OR neonat% OR newborn% OR infant OR child% OR toddler OR babies OR baby OR kids Searched the following terms individually, then excluded duplicates: paediatric, pediatric, child, children, kids, 20

29 adolescent, infant, newborn, toddler, baby, babies, neonate. Netherland s Trial Register ( egister.nl) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in the Netherlands Searched the following terms individually, then excluded duplicates: child, infant, paediatr, pediatr, adolescent, toddler, baby, babies, neonate, newborn. Registers included in Pandolfini and Bonati (2009) (21), but with incomplete data: Sri Lanka Clinical Trials Registry ( lk/) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in Sri Lanka Now possible to search this registry using the following terms individually, then excluded duplicates: paediatric, pediatric, child, children, kids, adolescent, infant, newborn, toddler, baby, babies, neonate. National Organisatio n for Rare Disorders (NORD) register ( Trials currently recruiting patients with rare diseases Database no longer appears to be functional 21

30 diseases.or g/) Roche ( e- trials.com/ Registry of Phase I-IV drug trials sponsored by Roche Not possible to search this registry, or quantify the number of included studies. ) UK pharm. industry (ABPI) ( org.uk) Was used to register trials sponsored or conducted by UK members of the ABPI Database no longer in existence. Registers not included in Pandolfini and Bonati (2009) (21): CRiS (Republic of Korea) (cris.nih.go. kr) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in South Korea Under the age of 18 filter DRKS (Germany) (drksneu.uniklini k- freiburg.de /drks_web /) Hospital, university and pharmaceutical research. Observational, prognosis and interventional studies. All studies are recruiting, but not necessarily hosted in Germany pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids EudraCT (EU) (eudract.e ma.europa. Hospital, University and Pharmaceutical research. Observational and interventional studies. All Using age range limit under

31 eu) studies have at least one investigator based in the EU. IFPMA Clinical trials portal (clinicaltrial s.ifpma.org /) Trials conducted by the pharmaceutical Industry. Observational and interventional studies. IRCT (Iran) ( r/) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in Iran. Pan African CTR ( r.org/) Hospital, university and pharmaceutical research. Interventional studies only (RCT or CCT). All studies are recruiting, but not necessarily hosted in Africa. ReBec (Brazil) ( iosclinicos. gov.br)** Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in Brazil pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids? ? The search software on the website was not functional and s to the registry received no response. 23

32 RPCEC (Cuba) (registrocli nico.sld.cu) UK Clinical Research Network: Portfolio Database (UK) (public.ukc rn.org.uk) UK Clinical Trials Gateway (UK) ( g.nihr.ac.uk ) UMIN-CTR (Japan) ( n.ac.jp/ctr/ ) Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in Cuba. Hospital, university and pharmaceutical research. Observational and interventional studies. Includes studies in set up, recruiting and closed studies. To register, all studies have to meet set criteria for portfolio adoption. NHS, university and pharmaceutical research. Observational and interventional studies. Hosted, but not limited to UK studies. Currently recruiting trials. Hospital, university and pharmaceutical research. Observational and interventional studies. All studies are recruiting, but not necessarily hosted in Japan Age limit < 18 years pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids : Age limit <18 years. 2017: Searched the following terms individually, then excluded duplicates: paediatric, pediatric, child, children, kids, adolescent, infant, newborn, toddler, baby, babies, 24

33 neonate. WHO - ICTRP pediatr* OR paediatr* OR adolesc* OR neonat* OR newborn* OR infant OR child* OR toddler OR babies OR baby OR kids Total (CCT: Controlled clinical trial; RCT: Randomised controlled trial) **Data not included in final results Some caution is required in interpreting the data presented in Table 3 for individual registries, as many studies have been entered into multiple databases. [Searches completed and data compiled by Dr Matthew Hyde, Imperial College London] 25

34 3.7 Queen s Anniversary Prizes The Prizes, one of the most prestigious awards in higher education, are part of the honours system and are awarded every two years by the Queen on the Prime Minister s advice. UK universities and colleges were invited in October 2016 to submit entries on any aspect of their work for assessment against the scheme s criteria of excellence, innovation and practical benefit, to the institution, UK education, UK economy, society and public benefit in general. In total, 21 UK universities and colleges of further education were recognised in 2017, seven of which are funded by the research councils. The awards include recognition of child health research, e.g. the Infant Sleep Information Source at Durham University funded by Economic and Social Research Council, the University of Edinburgh MRC Centre for Reproductive Health that has helped reduce stillbirths, and work at Cardiff University on the diagnosis and treatment of visual deficits in children with Down Syndrome. The RCPCH Is pleased to see high quality representation of children s research in these prestigious awards. 3.8 Children s research funding Turning the Tide [1,2] reported that total UK public and charitable medical research expenditure in the 2010/2011 financial year was 2.2 Billion, of which approximately 5% was directed at children s research. To ascertain whether there has been any change over the last five years, information on research expenditure was sought from UK funding bodies and charities (Table 4). Only data for organisations that provided figures for financial years 2012 to 2017 have been included in order that current research expenditure can be compared to that published in Turning the Tide. On this basis, overall research expenditure has increased to 2.8 Billion in 2015/16 of which 154 Million was directed at children s studies. The proportion of total expenditure on child health research rose to 8.5% in , but then fell year on year to 5.4% in European Commission child health research funding A search was carried out of the European Commission s Community Research and Development Information Service website to ascertain the amount of funding received by the UK. The search was limited to all Horizon 2020 projects which were either Coordinating Actions or Research Innovation Actions, using the search terms paediatr* and child*, from to The search identified 40 child health related projects with funding contributions from the EU, of which 32 had UK involvement. The total EU contribution to the 32 projects was just over 200 Million with 64 Million (32%) directed to the UK. The UK is the coordinator for 14 of the 32 projects. 26

35 Table 4 UK annual total public and charitable research expenditure, and proportion for child health research, to Charity Total research spend ( Million) Total children s research spend ( Million) % of total for child health research Total research spend ( Million) Total children s research spend ( Million) % of total for child health research Total research spend ( Million) Total children s research spend ( Million) % of total for child health research Total research spend ( Million) Total children s research spend ( Million) % of total for child health research Total research spend ( Million) Total children s research spend ( Million) % of total for child health research NIHR CCF % % % % % NIHR NETS % % % % % Medical Research Council Biotechnology and Biological Sciences Research Council % % % % % % % Arthritis Research UK % % % % % British Heart Foundation % % % % % British Lung Foundation % % % Cancer Research UK Diabetes UK % % % % % % % % % Epilepsy Research Meningitis Research Foundation Wellcome Trust % % % % % % % % % % % % % % % Total % % % % 2, % 3 Data presented are not directly comparable with the 2012 Turning the tide report as some organisations have made minor adjustments to previously reported figures 27 27

36 3.9 Research publications Contributions by NHS paediatricians In Turning the Tide a third of consultant level paediatricians reported authoring one or more peer reviewed papers in the two-year period from 2009 to The 2015 RCPCH survey of research participation found that 35% of consultant paediatricians had authored one or more peer reviewed papers during the two-year period up to May 2015 [22]. On average, men authored almost twice as many publications as women (4.0 versus 2.2) and sub-speciality paediatricians an average of 5.4 publications compared with 1.2 by general paediatricians Publications in the top biomedical, medical and paediatric journals Using a list of UK paediatric consultants from the RCPCH 2015 Medical Workforce Census [23], a systematic search was carried out of the PubMed database to identify the number of paediatric consultant publications between 2010 and The analysis was undertaken using the Pubmed Application Programming Interface ( and R (version 3.4.0) with the XML package (version ). An author with the same name as a UK consultant paediatrician was identified for 659,266 PubMed records. Of these, 70,050 were identified as originating from an institution with a UK address for the corresponding author. Of the UK publications, 551 (mean 92 per year) were published in one of the top 10 biomedical journals, 874 (mean of 146 per year) in the top 10 medical journals, and 207 (mean of 35 per year) in one of the top 10 paediatric journals (based on 2011 impact factors published by Thomson Reuters; table 5) (Table 6). There has been an increase in the annual number of publications in the top 10 paediatric journals from 30 in 2010 to 48 in 2015, but no substantial change in the number of publications in the top 10 biomedical and medical journals. 28

37 Table 5 Thomson Reuters 2011 top 10 biomedical, medical and paediatric journals Top 10 biomedical journals Top 10 medical journals Top 10 paediatric journals CA Cancer J Clin N Eng J Med J Am Acad Child Adolesc Psychiatry N Engl J Med Lancet Pediatrics Ann Rev Immunol JAMA Arch Pediatr Adolesc Med Nat Rev Mol Cell Biol Ann Intern Med J Pediatr Lancet PLos Med Dev Disabil Res Rev Nat Rev Genet BMJ Semin Fetal Neonatal Med. Nat Rev Cancer Arch Intern Med Pediatr Infect Dis J Nature CMAJ J Adolesc Health Nature Genet BMC Med Pediatr Crit Care Med Ann Rev Biochem Cochrane Database Syst Rev Arch Dis Child Fetal Neonatal Ed 29

38 Table 6 Total research publications from UK institutions that include a consultant paediatrician in the authorship, by year, from 2010 to All Years Total UK publications Total UK publications excluding reviews Publications from UK institutions including reviews Publications from UK institutions Publications from UK institutions in top 10 biomedical journals Publications from UK institutions in top 10 medical journals UK publications in top 10 paediatric journals Total reviews Reviews from UK institutions in top 10 biomedical journals Reviews from UK institutions in top 10 medical journals Reviews from UK institutions in top 10 paediatric journals excluding reviews [Searches and data compiled by Dr Andrew Prayle, University of Nottingham] 30

39 3.9.3 Publications involving children s studies A series of systematic searches of PubMed were carried out covering the years to determine the total number of papers in each year, the total number of studies involving adults and the total number involving children. The searches were independently tested (using a Boolean Search) to ensure they maximised the number of hits returned. The results are shown in Figure 2, as Children (PubMed Boolean Search) and show strong concordance, albeit a slightly higher number of publications, than the results obtained by using the PubMed children s study search. This shows that over the last 15 years the proportion of child to adult studies has remained unchanged at around third. Figure 2 Number of publications in children and adults in PubMed from 2002 to [Searches completed and data compiled by Dr Matthew Hyde, Imperial College London] 3 Figures are not directly comparable to Turning the Tide, as additional studies have been retrospectively indexed. 31

40 3.9.4 Publications in Archives of Disease in Childhood Archives of Diseases in Childhood is the principle journal of the RCPCH. This international peer review journal has an independent editor and editorial board. Established in 1926, the journal aims to keep paediatricians and others up to date with advances in the prevention, diagnosis and treatment of childhood diseases, and informed on advocacy issues. The overall acceptance rate from 2010 to 2015 for papers submitted to Archives of Disease in Childhood by UK paediatricians has remained relatively constant at around 50% (Table 7). Table 7 Submissions to Archives of Disease in Childhood and acceptance rates in 2005, 2010 and Total submissions UK submissions Rest of EU submissions Non-EU Europe UK acceptance rate (%) Rest of EU acceptance rate (%) Non-EU Europe acceptance rate (%) EU excluding UK National guidelines The National Institute of Health and Care Excellence (formerly Health and Clinical Excellence) (NICE) was established in 1999 with the aim of producing evidence-based guidelines and ending variation by geographical location in the delivery of treatments across the UK. Over the years, the remit of NICE has broadened in parallel with a change to becoming a non-departmental public body in Turning the Tide highlighted the limited evidence base for children s health care and the resulting predominance of consensus-based guidelines. Paediatric specific clinical and public health guidelines have fluctuated year on year but the number of paediatric Quality Standards has increased (Table 8). 32

41 Table 8 National Institute of Health and Care Excellence, outputs Clinical guidelines Total Paediatric-specific Paediatrics included Wholly nonpaediatric Public Health Guidelines Total Paediatric-specific Paediatrics included Wholly nonpaediatric Social Care Guidelines Total Paediatric-specific Paediatrics included Wholly nonpaediatric Quality Standards Total Paediatric-specific Paediatrics included Wholly nonpaediatric (The figures in this table represent new guidelines developed; updates of existing guidance are not included) 33

42 3.10 Conclusions BRC and CRF have strengthened opportunity for the translation of biomedical research. Harnessing the potential of developmental biology, children s sciences and life-course research would also bring substantial benefit to the UK population, no less children. The RCPCH would welcome increased representation of child health research in NIHR BRC and CRF. Recruitment of children to studies in the last five years has increased, as have publications by UK paediatricians in the world s top ten paediatric journals. The number of publications by UK paediatricians in the top 10 biomedical and medical journals has remained steady. A worrying finding is that men appear to author almost twice as many publications as women, though the reasons are unknown. Globally, the number of research publications relating to children s studies has increased in absolute terms but has remained constant as a proportion of all clinical studies. The proportion of funding awarded to child health by the research councils and major charities has also remained steady over the past five years. Taken as a whole, these findings are testament to the strong contribution of UK paediatricians to research, despite the current pressures facing the NHS. The findings also emphasise the necessity of addressing barriers to research involvement, and strengthening the competitiveness of child health researchers in order to assist them in securing a larger proportion of research council and charity awards. 34

43 4 The paediatric clinical academic workforce 4.1 The senior clinical academic workforce The RCPCH Medical Workforce Census [23] identified 168 clinical academic consultantlevel paediatricians (professors, readers and senior lecturers) in the UK in 2015 (Table 9). This represents 4.2% of the total consultant-level workforce compared with 3.8% in 2013, 4.6% in 2011 and 9.6% in The Medical Schools Council reported similar numbers, identifying 153 Full Time Equivalent academic consultant paediatricians in 2016 [24]. Table 9 4 Whole time equivalent professors, readers and senior lecturers from 2001 to 2015 [RCPCH 2015 Medical Workforce Census] Professors Readers Senior lecturers Total academic consultants NHS consultant involvement in research In 2011, the RCPCH conducted a survey [25], of all paediatric consultant and Staff, Associate Specialist and Specialty (SASS) doctors recorded in the 2009 workforce census achieving a response rate of 67%. Of respondents, 66% of consultant-level paediatricians had no allocated Programmed Activities (PA; representing 4 hours per week) for research, 26% had one PA or less, and 7% had 1.5 to 5 PA. Only 1%, all of whom hold academic appointments, reported receiving more than 5 research PA. The average number of research PA in consultant job plans was 0.5. The survey was repeated in 2015 [22]. All consultant and SASS doctors recorded in the RCPCH 2013 workforce census [26] and any new holders of a Certificate of Completion of Training (CCT) in paediatrics up to May 2015 were invited to participate. The response rate was 44% and was representative of the paediatric workforce with regard to sex, 4 Data are presented from the 2015 RCPCH medical workforce census and are may not be directly comparable to data reported in the 2011 Turning the Tide report due to retrospective confirmation of figures. 35

44 grade and location of work. The average number of research PA in consultant job plans was 0.4 (Figure 4), however the average number of research PA actually worked was 0.7. Though no research PA were reported by 80%, research involvement was reported by 49%. The Royal College of Physicians (London) reported similar findings. Over , a mean of 0.6 contracted PA was identified for academic work [27]. In 2015, the Royal College of Physicians also conducted a survey specifically focusing on research. This showed that doctors across all adult specialities and all career stages spent an average of 4.7 hours per week on research activities [28]. A substantial number of the RCPCH 2015 survey respondents (89%) reported having received no grants for research [22]. Awarders of the largest number of grants were local research charities (82), followed by national research charities (73) and the NIHR (54). Over a third of grant holders (127) were in receipt of grants totalling 200,000 or more from a single awarding body. Approximately 42% were in receipt of small grants i.e. less than 100,000. Only a minority of paediatric consultants were involved in PhD supervision (7.6%); 88% reported having never undertaken any PhD supervision. In 2017, the RCPCH surveyed trainees who obtained a paediatric CCT in 2015 or who obtained a place on specialist register through the Certificate of Eligibility for Specialist Registration route. Three hundred and twenty-four trainees were identified, of whom 179 responded (55%). The purpose of the survey was to identify career intentions and working patterns. Of 128 respondents, 47% (60/128) stated they would like to be undertaking more academic/research work but only 23% (29/128) expected to do so (Table 10). 36

45 Table 10 Career development expectations Career development statement Expect career to develop Would like career to develop Number % Number % I will be carrying out more specialist work I will be undertaking more medical education work I will be more involved in trust/ service management I will be undertaking more academic/research work I will be undertaking roles for the RCPCH I will be undertaking less resident shift working I will be undertaking less Direct Clinical Care I will be working in a different area of paediatrics I will not be working in the UK I will be working in a different medical specialty I will not be working in medicine

46 Figure 4 Programmed activities for research in job plan and actually worked (consultants only) [RCPCH 2015 research participation survey] 4.3 Research fellowships Clinical academics bridge practice and research and fellowships are critical to support career progression. The MRC carried out a survey on behalf of 13 research funders, the Medical, Dental and Veterinary Schools Councils and the Association of Medical Research Charities, on personal fellowship awards 5 active in March 2017 [29]. Data include individuals awarded clinical and health research fellowships who were medically qualified, dentists, nurses and midwives, veterinarians, allied and other health professionals, and non-health professionals. This provided a snapshot of fellowship funding live in March 2017 across England, Wales, Scotland and Northern Ireland. Overall, 2840 active fellowships were identified across all medical specialities and career stages from pre-doctoral to senior academic appointments, with a 71% increase from 2009, equating to 1180 additional fellowships. There was a marked increase in the award of early career fellowships, possibly due to the Academic Clinical Fellowships established by the NIHR. The Integrated Academic Training pathway was established in 2006 and includes Academic Clinical Fellowships 5 Fellowship awards supporting the salary of the recipient (the fellow), usually for between 1 and 5 years; most fellowships pay for 100% of the fellow s contracted working time to enable a consolidated period to be focussed on research. 38

47 (ACF) and Academic Clinical Lectureships (ACL), coordinated by the NIHR Trainees Coordinating Centre. In 2017, the Centre undertook a strategic review of training [30]. This recommended the integration of all current academic training and higher career personal awards into a new academic structure with the creation of a new entity, the NIHR Academy. The NIHR Academy will replace the NIHR Trainees Coordinating Centre to host all academic training and career development activity. NIHR trainees will become NIHR academy members and criteria will be established for individuals who are not fully funded by NIHR to be eligible for Academy membership as associates, allowing them to become part of the research training ecosystem. The Integrated Academic Training pathway is a tool for expanding capacity in priority areas. Allocation by formula, where decisions about speciality are made at the local partnership level, is meant to support academic strengths locally but risks lowering competitiveness. Hence, the review recommended that the balance between the allocation of posts by formula and competition should be modified to ensure a closer link with NIHR strategic priorities, as well as addressing underrepresented areas and future health challenges identified through consultation. For competitive posts, themes have been developed where research is linked to complex challenges [31]. Data over the period 2010/ /17 from the NIHR Trainees Coordinating Centre (Table 8) highlights an average of 254 ACF appointed each year. The number of annual paediatric ACF awards has fallen from 31 to 16, though the proportion following an abrupt drop from 13% to 7% over 2010/11 to 2011/12 has remained steady. The number of ACL appointed each year has averaged 89 since 2010/11 with the number of paediatric awards ranging from two to nine. Data for 2016 provided by the Medical Schools Council shows the number of paediatric lecturers to have fallen to 23.2 WTE (personal communication). 39

48 Table 11 National Institute for Health Research Academic Clinical Fellowship (ACF) awards and Academic Clinical Lectureship (ACL) awards, 2010/11 to 2016/ / / / / / / /17 Mean Paediatric ACF posts Total ACF posts Percentage of ACF posts awarded to paediatrics Paediatric ACL posts Total ACL posts Percentage of ACL posts awarded to paediatrics *Including paediatric cardiology and surgery 4.4 Medically qualified researcher progression The 2017 MRC survey [29] reported that the number of pre-doctorial (awards supporting initial research experience; i.e. a Masters course or NIHR ACF award) and doctoral awards (awards supporting a PhD or other higher degree) supported by the UK funders partnering in this survey has increased since The total number of awards supporting the initial post-doctoral career stage has almost doubled since 2009 demonstrating an improvement in the breadth of routes available to individuals to access academic training post-phd. However, the survey identified the need for more support for post-doctoral fellows, as there has been a 13% decline in awards supporting fellows to establish an independent research career. In 2009, 198 live awards were identified, compared with 172 in 2017, suggesting this represents an increasing bottleneck in the pathway to a senior clinical academic position. In total, 2149 medically qualified fellows across all career stages were identified, compared to 1343 in Paediatrics and child health was shown to have the fifth highest number of medically qualified fellows across all medical specialities and all career stages [29], and has featured within the top ten since 2009 (Figure 5). 40

49 Figure 5 Distribution of medically qualified fellows by medical speciality and career stage (NIHR ACF, In-Practice Fellowships (IPF) and ACL) in 2017 [Graph supplied by Medical Research Council

50 Figure 6 shows the change in the proportion of fellowships across specialities, including pre- doctorial (ACF, IPF, Masters) and ACL awards. There has been a small decline in the proportion of fellowships in paediatrics and child health between 2009 and Figure 6 Change in the proportion of fellowships by medical speciality in 2009 and 2017, including pre-doctorial and ACL awards [Graph supplied by Medical Research Council